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• W3080948614 endingPage "939" @default.
• W3080948614 startingPage "921" @default.
• W3080948614 abstract "The lung has long been a target for gene therapy, yet efficient delivery and phenotypic disease correction has remained challenging. Although there have been significant advancements in gene therapies of other organs, including the development of several ex vivo therapies, in vivo therapeutics of the lung have been slower to transition to the clinic. Within the past few years, the field has witnessed an explosion in the development of new gene addition and gene editing strategies for the treatment of monogenic disorders. In this review, we will summarize current developments in gene therapy for cystic fibrosis, alpha-1 antitrypsin deficiency, and surfactant protein deficiencies. We will explore the different gene addition and gene editing strategies under investigation and review the challenges of delivery to the lung." @default.
• W3080948614 created "2020-09-01" @default.
• W3080948614 creator A5027748326 @default.
• W3080948614 creator A5085725734 @default.
• W3080948614 date "2020-09-01" @default.
• W3080948614 modified "2023-09-24" @default.
• W3080948614 title "New Directions in Pulmonary Gene Therapy" @default.
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